Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

  • End date
    Dec 29, 2022
  • participants needed
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 29 April 2022
stem cell transplantation
bone marrow transplant
cell transplantation
bone marrow procedure
langerhans cell histiocytosis
lymphoproliferative disorder
x-linked lymphoproliferative disease
allogeneic hematopoietic stem cell transplant
wiskott-aldrich syndrome
lymphoproliferative disease
hemophagocytic lymphohistiocytosis
omenn's syndrome
treatment guideline
chronic granulomatous disease
chediak-higashi syndrome
primary immune deficiencies
granulomatous disease
reticular dysgenesis
primary immunodeficiency
severe combined immunodeficiency
omenn syndrome
griscelli syndrome
cd40 ligand


This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.


Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans:

Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen

Condition SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
Treatment cyclophosphamide, Stem cell transplantation, busulfan, melphalan, MESNA, Alemtuzumab 0.3 mg, Fludarabine phosphate 40 mg, Alemtuzumab 0.2 mg, Fludarabine phosphate 30 mg
Clinical Study IdentifierNCT01652092
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on29 April 2022


Yes No Not Sure

Inclusion Criteria

Diagnosis of immunodeficiency or histiocytic disorder including the following
Severe combined immunodeficiency (SCID - all variants)
Second bone marrow transplant (BMT) for SCID (after graft rejection)
Omenn's Syndrome
Reticular dysgenesis
Wiskott-Aldrich syndrome
Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
Hyper IgM Syndrome (CD40 Ligand Deficiency)
Common variable immunodeficiency (CVID) with severe phenotype
Chronic Granulomatous Disease (CGD)
Other severe Combined Immune Deficiencies (CID)
Hemophagocytic Lymphohistiocytosis (HLH)
X-linked Lymphoproliferative Disease (XLP)
Chediak-Higashi Syndrome (CHS)
Griscelli Syndrome
Langerhans Cell Histiocytosis (LCH)
Acceptable stem cell sources include
HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
HLA identical or up to a 1 antigen mismatched unrelated BM donor
Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
Double unrelated umbilical cord blood units that are
up to 2 antigen mismatched to the patient
up to 2 antigen mismatched to each other
minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
Age: 0 to 50 years
Adequate organ function and performance status

Exclusion Criteria

pregnant or breastfeeding
active, uncontrolled infection and/or HIV positive
acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Clear my responses

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